Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis

Completed · Phase 1/Phase 2

Conditions studied: Leber Congenital Amaurosis

In brief

The purpose of the study is to evaluate the safety and efficacy of an adeno-associated virus vector expressing RPE65 in patients with Leber congenital amaurosis caused by mutations in the RPE65 gene. Funding Source - FDA OOPD

Key facts

Study ID

NCT00749957

Run by

Beacon Therapeutics

People needed

12

Starts

2009-06-17

Expected to finish

2017-09-22

Last updated by the study team

2017-12-28

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Retinal disease consistent with a diagnosis of Leber congenital amaurosis and documented mutations in the RPE65 gene (including null mutations and mutations that code for abnormal RPE65 protein);
• At least 6 years of age;
• Good general health without significant physical examination findings or clinically significant abnormal laboratory results;
• Able to perform tests of visual and retinal function;
• Visual acuity not better than 20/60 and not worse than hand motion in both the treated eye and the fellow eye;
• Visible photoreceptor (outer nuclear) layer on a standard optical coherence tomography (OCT) scan;
• Acceptable hematology, clinical chemistry and urine laboratory parameters;
• For females of childbearing potential, a negative pregnancy test at screening and at baseline, and agreement to use effective contraception for 12 months after administration of rAAV2-CB-hRPE65, for sexual activity that could lead to pregnancy;
• For males of reproductive potential, agreement to use effective contraception for 12 months after administration of rAAV2-CB-hRPE65, for sexual activity that could lead to pregnancy

You may not qualify if…

• Pre-existing eye conditions that would preclude the planned surgery or interfere with interpretation of study endpoints or complications of surgery (e.g. glaucoma, corneal or lenticular opacities, or history or retinal detachment);
• Presence of epiretinal membrane on OCT;
• History of immunodeficiency or other medical conditions that might increase the risk of rAAV2-CB-hRPE65 administration;
• Use of anticoagulants or anti-platelet agents within 7 days prior to study agent administration;
• History of allergy or sensitivity to medications planned for use in the peri-operative period;
• For females of childbearing potential, a positive pregnancy test at screening or baseline (within 2 days before rAAV2-CB-hRPE65 administration);
• Females who are breast feeding;
• Use of any investigational agent, or systemic corticosteroids or other immunosuppressive drug(s), within 3 months prior to enrollment;
• Prior receipt of any AAV gene therapy product;
• Any condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.

Where it is running

• University of Massachusetts Medical School — Worcester, Massachusetts, United States
• Casey Eye Institue, Oregon Health & Science University — Portland, Oregon, United States

Full record on ClinicalTrials.gov

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